Charcot-Marie-Tooth Disease, Type Ia

COLUMBUS, Ohio, Jan. 15, 2024 /PRNewswire/ -- Andelyn Biosciences, Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has partnered with Armatus Bio to accelerate manufacturing of their gene therapy treatment for Charcot-Marie-Tooth Type 1A (CMT1A), a rare genetic neurological disease associated with independence-limiting disability and risk of fatal complications that has no approved therapies today. The partnership will seek to maximize program efficiency and speed to clinical studies of this novel, precision approach. As part of the agreement, Armatus will leverage Andelyn's extensive experience in adeno-associated virus (AAV) production and its proprietary suspension platform for development activities, plasmid manufacturing, and viral vector toxicology and GMP clinical manufacturing. The partnership strengthens the working relationship and offers great hope for patients suffering from neuromuscular disease. Both organizations, located in the well-established and growing biotech hub of Columbus, Ohio, are aligned in their belief that the rise of gene therapies will continue to advance human health. Matt Niloff, Chief Commercial Officer at Andelyn Biosciences said, "We are extremely moved by Armatus Bio's commitment to address the urgent unmet needs in CMT1A and are impressed by its innovative therapeutic technology for the disease. We look forward to scaling up the process in our program-specific, configurable suspension platform, and accelerating the therapy into the clinic with the highest degree of quality." "Andelyn has demonstrated its strong capabilities in manufacturing complex genetic medicines with high quality and consistency, which will be critical to accelerating our development efforts," said Brian Price, PhD, Chief Technical Officer of Armatus. "We look forward to collaborating with Andelyn on this program as we work toward supporting this population that urgently awaits innovative solutions." From its time-tested experience, Andelyn is proud to offer its clients exceptional development, plasmid production, and quality manufacturing at its three Columbus, Ohio facilities. Andelyn is advancing client discoveries and manufacturing life-altering cell and gene therapies for rare and prevalent diseases. About Andelyn Biosciences, Inc. Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn's deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of three Columbus, Ohio facilities, Andelyn supports its clients in developing curative cell and gene therapies from concept through plasmid development and manufacturing, process development, and cGMP clinical and commercial manufacturing. Andelyn's versatile capabilities include cGMP manufacturing capacity for both adherent and suspension processes up to a 2,000-liter capacity. An advanced digital model, quality system, full regulatory support and supply chain vertical integration help Andelyn accelerate the development and manufacturing of its clients' innovative cell and gene therapies. For more information, visit andelynbio.com. About Armatus Bio Armatus Bio is a privately held late preclinical stage biotechnology innovator leveraging vectorized RNAi to address urgent unmet medical needs in genetically-driven neurological diseases. Based in Columbus, Ohio, the company is led by a seasoned team with expertise in drug development and delivery, and partnered with world renowned experts in vector biology, genomics, and neurology. The company's lead candidate for Charcot Marie Tooth Type 1A (CMT1A) has received Orphan Drug Designation and Rare Pediatric Drug Designation, and is advancing toward IND-enabling studies. The company is also developing a preclinical vectorized RNAi asset for the rare neuromuscular disease Facioscapulohumeral Muscular Dystrophy (FSHD). SOURCE Andelyn Biosciences, Inc.

Pharnext hopes incoming data from a phase 3 trial run by its Chinese partner Tasly will offer some better news. Less than a year after Pharnext dropped its Alzheimer’s program over cost concerns, the French biotech has seen its lead program fail a phase 3 trial in a rare neurological disease. The company is developing PXT3003 in Charcot-Marie-Tooth disease type 1A, an inherited genetic disorder that affects the peripheral nerves, resulting in muscle weakness and potentially sensory loss. The 387-patient trial did not show that the drug was able to improve functional motor disability as measured by the Overall Neuropathy Limitation Scale (ONLS), the company revealed in a Dec. 11 release. Pharnext blamed an “unexpected” improvement in the disease among the placebo cohort, which it said “complicates the interpretation of the results.” “It also confirms what the medical literature as a whole has been saying for many years, namely that ONLS is undoubtedly a relevant endpoint over the long term, but not over such a short period of time as a clinical study,” the company claimed in the release. Trying to see the bright side, the biotech said there had been “no deterioration” of the condition among the patients who received the drug. “This suggests that PXT3003 might stabilize the condition of patients, which is an important consideration for a disease where progression is generally inevitable,” it concluded. PXT3003 is a combination of the approved meds baclofen, naltrexone and sorbitol. The oral drug is designed to downregulate the overexpression of the PMP22 protein in order to improve neuronal signaling in dysfunctional peripheral nerves. Pharnext hopes incoming data from a phase 3 trial run by its Chinese partner Tasly will offer some better news. In the meantime, the company “plans to continue analyzing the data, particularly in collaboration with potential partners for licensing or acquisition of PXT3003.” “Although the results we are sharing today are not exactly what we had hoped for, they are nonetheless very promising,” Pharnext’s Manager Hugo Brugière said in the release. “We are now going to make the most of all the data we have accumulated over the last 10 years, including our two phase 3 studies and our 6-year extension study, which tend to demonstrate a beneficial effect on patients.” “I remind that, today, PXT3003 is the only ray of hope for all CMT1A patients, and that no other drug candidate is currently in an advanced stage,” Brugière added. “It seems to me, therefore, that efficacy results for a risk-free drug could support an application for regulatory approval and market authorization. This is for us the final stage, which we will move forward with the patient community." Investors didn’t appear to see much cause for optimism, sending the company’s share price plunging 68% on Monday morning. However, with the company’s stock already sitting at below one euro cent, the difference was negligible in practicable terms. Pharnext’s stock crashed back in February, when the biotech walked away from its potential Alzheimer’s asset, dubbed PXT864, arguing that the encouraging data produced to date weren't enough to lead to commercialization in the “short or medium term.”  Instead, Brugière said at the time that the company would search for a partner to take the clinical baton.  It left Pharnext even more reliant on PXT3003. The company said in October that it had received potential international licensing offers worth over 250 million euros for the drug, although it remains to be seen whether these as-yet-undisclosed deals will be impacted by today’s results.

FIRST INVESTMENT FROM CMTA'S NEW VENTURE PHILANTHROPY ARM GLENOLDEN, PA / ACCESSWIRE / November 16, 2023 / The Charcot-Marie-Tooth Association (CMTA) today announced that it participated in the Seed Series Extension syndicate for Armatus Bio, Inc. (Armatus). Armatus is an emerging Biotechnology company that is advancing a unique gene therapy clinical candidate to target CMT type 1A, the most common form of the disease. Charcot-Marie-Tooth disease is an inherited peripheral neuropathy, principally affecting the long nerves of the hands and feet. Currently there are no FDA-approved therapies for any type of CMT. Thomas Dubensky, Ph.D., a CMTA board member commented "The CMTA is very excited to facilitate the advancement of novel therapies to be ultimately evaluated in patients living with all types of CMT. Armatus is the CMTA's first investment from its new venture philanthropy arm, which will make selected early-stage investments in Biotech companies developing unique and promising therapies against all types of CMT." Dr. Dubensky went on to say that "the venture philanthropy arm will work in tandem with STAR (Strategy to Accelerate Research), the arm of CMTA which is the largest private funder of CMT research in the world." Rachel Salzman, DVM, CEO of Armatus, commented, "Armatus is extremely grateful to the CMTA for its support, and we look forward to their continued advice and counsel as we advance ARM-101." Dr. Salzman went on to say, "Our ARM-101 development studies are particularly important for the CMT community because the data generated are designed to answer critical questions about how we can effectively deliver genetic medicines to the right parts of the body to meaningfully address this disease." She added, "As we accelerate development of ARM-101 and gain further evidence that our vectorized RNAi can normalize PMP22 in Schwann cells as a means of enabling human trials, we believe this therapy has the potential to provide benefit to the thousands of people living with CMT1A in the U.S. and around the world." Armatus Bio, based in Columbus, Ohio, is an emerging biotechnology company that is collaborating with renowned gene therapy experts to build a pipeline of novel therapeutic candidates designed to overcome the limitations of today's approaches and propel the next generation of genetic medicines. ABOUT THE CHARCOT-MARIE-TOOTH ASSOCIATION The Charcot-Marie-Tooth Association (CMTA) is the largest philanthropic funder of CMT research worldwide. The CMTA's Strategy to Accelerate Research (STAR) brings the best CMT researchers, clinicians, and experts in therapy development together with patients, pharmaceutical and biotechnology companies to expedite the development of treatments for CMT. Since 2008, the CMTA has invested more than $23.5 million via STAR. The CMTA also improves quality of life for CMT families by offering educational programs and materials, patient and professional conferences, support through youth programs, its branch system and global clinical Centers of Excellence. To learn more visit cmtausa.org and follow the CMTA on Instagram, Facebook, Twitter, TikTok, LinkedIn, and YouTube. Contact Information Marcia Semmes Media Contact, Charcot-Marie-Tooth Association marcia@cmtausa.org SOURCE: Charcot-Marie-Tooth Association View source version on accesswire.com: